Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

An Extension Study of AGT-181-102 Evaluating Safety and Glycosaminoglycans (GAGs) in Adult Patients With Hurler-Scheie or Scheie Syndrome Who Have Completed 8-Weeks of Dosing With AGT-181 in Study AGT-181-102

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02597114

Enrollment

Registered

2015-11-05

Start date

2015-11-30

Completion date

2018-08-02

Last updated

2018-09-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mucopolysaccharidosis I

Brief summary

AGT-181 is a fusion protein containing alpha-L-Iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously. This study is an extension of a safety and dose ranging study to obtain long term safety and exposure data, as well as information on the biological activity of the investigational drug

Interventions

DRUGAGT-181

intravenous infusion over 3-4 hours

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Masking description

Open Label

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Must have completed clinical trial AGT-181-102 * Voluntary written consent by patient or legally responsible representative * All women of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study. * Negative pregnancy test (females)

Exclusion criteria

* Refusal to complete screening evaluations. * Any medical condition or other circumstances that may significantly interfere with study compliance * Patient is pregnant or lactating * Clinically significant spinal cord compression, evidence of cervical instability. * Subject developed clinically relevant hypersensitivity to AGT-181

Design outcomes

Primary

Measure	Time frame
Number of participants with adverse events as a measure of safety and tolerability	104 weeks (2 years)

Secondary

Measure	Time frame
changes in urinary or plasma glycosaminoglycans (GAGs)	104 weeks (2 years)
change in liver size	104 weeks (2 years)
change in spleen size	104 weeks (2 years)
change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid	104 weeks (2 years)

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026